CY1122511T1 - Διαμορφωση της tmprss6 εκφρασης - Google Patents

Διαμορφωση της tmprss6 εκφρασης

Info

Publication number
CY1122511T1
CY1122511T1 CY20201100001T CY201100001T CY1122511T1 CY 1122511 T1 CY1122511 T1 CY 1122511T1 CY 20201100001 T CY20201100001 T CY 20201100001T CY 201100001 T CY201100001 T CY 201100001T CY 1122511 T1 CY1122511 T1 CY 1122511T1
Authority
CY
Cyprus
Prior art keywords
modulation
tmprss6 expression
modulating
antisense compounds
tmprss6
Prior art date
Application number
CY20201100001T
Other languages
English (en)
Inventor
Shuling Guo
Original Assignee
Ionis Pharmaceuticals, Inc.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Ionis Pharmaceuticals, Inc. filed Critical Ionis Pharmaceuticals, Inc.
Publication of CY1122511T1 publication Critical patent/CY1122511T1/el

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Steroid Compounds (AREA)

Abstract

Στο παρόν αποκαλύπτονται αντινοηματικές ενώσεις και μέθοδοι διαμόρφωσης TMPRSS6 και διαμόρφωσης ασθένειας, διαταραχής και/ή πάθησης συσσώρευσης σιδήρου σε ένα άτομο που έχει ανάγκη αυτής. Η ασθένεια συσσώρευσης σιδήρου σε ένα άτομο όπως αιμοχρωμάτωση ή β-θαλασσαιμία μπορεί να βελτιωθεί ή να προληφθεί με την χορήγηση αντινοηματικών ενώσεων που στοχεύονται σε TMRSS6.
CY20201100001T 2011-11-07 2020-01-02 Διαμορφωση της tmprss6 εκφρασης CY1122511T1 (el)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201161556755P 2011-11-07 2011-11-07
US201261710636P 2012-10-05 2012-10-05
PCT/US2012/063970 WO2013070786A1 (en) 2011-11-07 2012-11-07 Modulation of tmprss6 expression

Publications (1)

Publication Number Publication Date
CY1122511T1 true CY1122511T1 (el) 2021-01-27

Family

ID=48290517

Family Applications (1)

Application Number Title Priority Date Filing Date
CY20201100001T CY1122511T1 (el) 2011-11-07 2020-01-02 Διαμορφωση της tmprss6 εκφρασης

Country Status (6)

Country Link
US (4) US9725722B2 (el)
EP (2) EP2776564B1 (el)
CY (1) CY1122511T1 (el)
ES (1) ES2761343T3 (el)
HR (1) HRP20191883T1 (el)
WO (1) WO2013070786A1 (el)

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MX343008B (es) 2011-03-29 2016-10-21 Alnylam Pharmaceuticals Inc Composiciones y metodos para inhibir la expresion del gen de proteasa transmembrana, serina 6 (tmprss6).
IL293657A (en) * 2013-05-22 2022-08-01 Alnylam Pharmaceuticals Inc Preparations of tmprss6 irna and methods of using them
WO2016085852A1 (en) 2014-11-24 2016-06-02 Alnylam Pharmaceuticals, Inc. Tmprss6 irna compositions and methods of use thereof
CA2978100C (en) 2015-04-03 2023-10-17 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating tmprss6 expression
CN110177544A (zh) 2016-11-29 2019-08-27 普尔泰克健康有限公司 用于递送治疗剂的外泌体
JP7247167B2 (ja) * 2017-04-05 2023-03-28 サイレンス・セラピューティクス・ゲーエムベーハー 産物及び組成物
WO2021207072A1 (en) * 2020-04-07 2021-10-14 Mabwell Therapeutics Inc. Anti-tmprss6 antibodies and uses thereof
EP4330392A1 (en) 2021-04-26 2024-03-06 Alnylam Pharmaceuticals, Inc. Transmembrane protease, serine 6 (tmprss6) irna compositions and methods of use thereof
MX2023012834A (es) * 2021-04-27 2023-11-08 Silence Therapeutics Gmbh Inhibidor de la matriptasa-2 para el tratamiento de trastornos mieloproliferativos.
TW202400193A (zh) 2022-06-24 2024-01-01 丹麥商諾佛 儂迪克股份有限公司 抑制跨膜絲胺酸蛋白酶６（ｔｍｐｒｓｓ６）表現的組成物及方法
CN117136236B (zh) * 2022-09-29 2024-03-15 广州必贝特医药股份有限公司 用于抑制TMPRSS6基因表达的siRNA或其盐、药物及其应用

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Also Published As

Publication number Publication date
HRP20191883T1 (hr) 2019-12-27
ES2761343T3 (es) 2020-05-19
US20170073688A1 (en) 2017-03-16
US20180223289A1 (en) 2018-08-09
US10184125B2 (en) 2019-01-22
US9885047B2 (en) 2018-02-06
WO2013070786A1 (en) 2013-05-16
US20140309286A1 (en) 2014-10-16
EP3650544A1 (en) 2020-05-13
EP2776564A1 (en) 2014-09-17
US20190338288A1 (en) 2019-11-07
EP2776564B1 (en) 2019-10-02
EP2776564A4 (en) 2015-06-03
US9725722B2 (en) 2017-08-08

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